New hope for kids with rare liver disease: long-term drug study launched
NCT ID NCT05035030
First seen Jun 27, 2026 · Last updated Sep 01, 2026 · Updated 3 times
Summary
This study looks at the long-term safety and effectiveness of odevixibat in 70 people with Alagille syndrome, a rare genetic condition that affects the liver and other organs. The drug aims to reduce severe itching and improve bile flow. Participants who completed a prior 24-week study can join this extension to see how well the treatment works over time.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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62 people
The number who actually took part.
- Started
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Sep 2021
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: Cohort 1 : 1. Completion of the 24-week Treatment Period of Study A4250-012 2. Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study 3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study 4. Sexually active males and females must agree to use a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) from signed informed consent through 90 days after last dose of study drug. Cohort 2 : 1. Infant with clinically confirmed ALGS , ≤11 months of age at Study Day 1 2. Body weight ≥2 kg at Study Day 1 3. Gestational age ≥36 weeks. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required . 4. Signed parent/legal guardian informed consent. Exclusion Criteria: Cohort 1 : 1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy 2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012 3. Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study 4. Known hypersensitivity to any components of odevixibat Cohort 2 : 1. Patient with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following: 1. Biliary atresia of any kind 2. Progressive familial intrahepatic cholestasis (PFIC) 3. Benign recurrent intrahepatic cholestasis 2. Patient with a past medical history or ongoing presence of any other disease or condition known to interfere with the absorption, distribution, metabolism (specifically bile acid metabolism), or excretion of drugs in the intestine, including but not limited to, inflammatory bowel disease 3. Patient with past medical history or ongoing chronic diarrhea requiring intravenous fluid or nutritional intervention for treatment of the diarrhea and/or its sequelae 4. Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant chronic infection 5. Recent infection requiring hospitalization or treatment with parenteral anti-infective within 4 weeks of Study Day 1 or completion of oral anti-infective treatment within 2 weeks prior to the Screening Visit 6. Cancer diagnosis (except for basal cell carcinoma) 7. Chronic kidney disease with an impaired renal function and a glomerular filtration rate \<70 mL/min/1.73 m2 8. Patient with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to the Screening Visit 9. Patient has had a liver transplant, or a liver transplant is planned within 6 months of Study Day 1 10. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy 11. International normalized ratio (INR) \>1.4 (the patient may be treated with Vitamin K, and if INR is ≤1.4 at resampling the patient may be enrolled) 12. Serum alanine aminotransferase (ALT) \>10 × upper limit of normal (ULN) at Screening 13. Serum ALT \>15 × ULN at any time point during the last 6 months unless an alternate etiology was confirmed for the elevation 14. Total bilirubin \>15 × ULN at Screening 15. Patient suffers from uncontrolled, recalcitrant pruritic condition other than ALGS. Examples include, but not limited to, refractory atopic dermatitis or other primary pruritic skin diseases. 16. Patient exposed to alcohol or substance abuse in utero 17. Bile acid or lipid binding resins and medications that slow gastrointestinal motility 18. Patient has had investigational exposure to a drug, biologic agent, or medical device within 30 days prior to the Screening Visit, or 5 half-lives of the study agent, whichever is longer 19. Any other conditions or abnormalities which, in the opinion of the investigator may compromise the safety of the patient, or interfere with the patient participating in or completing the study
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Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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AOU Meyer
Florence, Italy
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Antenne pediatrique du CIC-Hopital Jeanne De Flandre
Lille, France
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Atrium Health Carolinas Medical
Durham, North Carolina, 27710, United States
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Azienda Ospedale University
Padova, 35128, Italy
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Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Charité - Universitätsmedizin Berlin
Berlin, 13353, Germany
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
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Children's Mercy Hospital and Clinics
Kansas City, Missouri, 64018, United States
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Childrens Medical Center of Dallas University of Texas Southwestern
Dallas, Texas, 75207, United States
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Cliniques Universitaires Saint-Luc Bruxelles
Brussels, 1200, Belgium
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Hassenfeld Children's Hospital at NYU Langone
New York, New York, 10016, United States
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Hopital Necker Enfants Malades
Paris, 75015, France
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Hôpital Femme Mère Enfant de Lyon
Bron, 69677, France
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Instytut Pomnik-Centrum Zdrowia Dzieck
Warsaw, 04-730, Poland
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Istanbul University Istanbul Medical Faculty Hospital
Istanbul, Turkey (Türkiye)
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Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
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King's College Hospital NHS Foundation Trust King's College Hospital Paediatric Research
London, SE5 9RS, United Kingdom
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Medizinische Hochschul
Hanover, 30625, Germany
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Monroe Carell Jr. Childrens Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
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New York-Presbyterian / Columbia University Irving Medical Center
New York, New York, 10032, United States
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Northwell Health System
New Hyde Park, New York, 11042, United States
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Oregon Health Science University School of Medicine
Portland, Oregon, 97239, United States
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Ospedale Pediatrico Bambino Gesu
Rome, 00165, Italy
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Rady Children's Hospital
San Diego, California, 92123, United States
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Riley Hospital for Children at IU Health
Indianapolis, Indiana, 46202, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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Texas Liver Institute
San Antonio, Texas, 78215, United States
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The Childrens Hospital at Montefiore Albert Einstein School of Medicine
The Bronx, New York, 10467, United States
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UCSF
San Francisco, California, 94158, United States
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Universitair Medisch Centrum Groningen
Groningen, 9713 GZ, Netherlands
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Universitatsklinik fur Kinder-und Jugendmedizin Tubingen
Tübingen, 72076, Germany
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University of Malaya Medical Center
Kuala Lumpur, 59100, Malaysia
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Wilhelmina Children's Hospital UMCU Utrecht
Utrecht, Netherlands
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New drug shows promise for babies with rare liver disorders
- New study tracks Real-World safety of odevixibat in rare liver disease
- Can a common cholesterol drug shrink skin lumps in kids with rare liver disease?
- New drug livmarli tracked for Long-Term safety in kids with rare liver disorders
- Liver drug maralixibat tested for Long-Term safety in rare disease patients
- Major study on rare childhood liver diseases halted